FMT for Autism

Overseen by Pat Levitt, Ph.D

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Waitlist Available

Sponsor: Children's Hospital Los Angeles

Trial Summary

What is the purpose of this trial?

The purpose of this study is to find out if transplant of fecal matter (stool), also known as fecal microbiota transplantation (FMT), from a healthy person into the intestines of children and young adults with Autism Spectrum Disorder (ASD). For this study children between the ages of 5-17years will be recruited over 2 years. Children will be recruited who receive an ASD diagnosis using the gold-standard Autism Diagnosis Observation Schedule -2 (ADOS-2) using module 1, 2 or 3 (none, limited or no moderate expressive language). Children diagnosed with these modules of the ADOS-2 may be at greater risk for GI disorders and rigid-compulsive behaviors. Additional assessment of rigid-compulsive behaviors and social communication will be done using the Repetitive Behavioral Scales-Revised (RBS-R) and Social Responsiveness Scale-2 (SRS-2), respectively. KBIT (the Kaufman Brief Intelligence Test) is used at baseline to obtain patient IQ. Total evaluation time is approximately 90 minutes. Following baseline symptom evaluation, a medical exam will be performed to determine whether each child is expressing specific GI symptoms. In addition, parents will fill out the Questionnaire for Pediatric Gastrointestinal Symptoms- Rome III (QPGS-III). Once an ASD diagnosis is confirmed, FMT treatment will be initiated, which typically occurs within 4-6 weeks of the initial diagnosis. Half 50% of the children (n=5) will receive the equivalent of 50 g of stools from a healthy donor into the jejunum through upper endoscopy and the other 50% off children (n=5) will receive Saline solution as Placebo control through upper endoscopy. Subjects will have a total of 5 visits within 24 weeks including phone call follow up on Day 7 after FMT.

Research Team

Sonia Michail, MD

Principal Investigator

Children's Hospital Los Angeles

Pat Levitt, Ph.D

Principal Investigator

Children's Hospital Los Angeles

Eligibility Criteria

This trial is for children aged 5-17 with Autism Spectrum Disorder (ASD) who have gastrointestinal symptoms. They must be diagnosed using specific autism and GI symptom assessments, need an upper GI endoscopy, and not have any severe sensory or motor problems, genetic syndromic disorders, significant renal/liver dysfunction, immunodeficiency conditions, or severe food allergies.

Inclusion Criteria

ADOS validated diagnoses of ASD

Questionnaires: RBS-2, KBIT, SRS, Rome III Version (QPGS- RIII), Ped QL, SSP

I am between 5 and 17 years old and have been diagnosed with autism.

Exclusion Criteria

You were born before 36 weeks of pregnancy.

Pregnancy: testing will be done on FMT day 0 for subjects with childbearing potential

I am capable of giving consent but choose not to.

Treatment Details

Interventions

• FMT (Fecal Microbiota Transplantation)

Trial Overview The study tests if fecal microbiota transplantation (FMT)—transferring stool from a healthy donor to the intestines—helps kids with ASD. Participants are randomly chosen to receive either FMT or a saline placebo through an endoscopy into the jejunum.

Participant Groups

Group I: Arm 2 Active Control 1 Intervention

Active Comparator: Donor Stool Transplant Arm 2 will get FMT (Fecal Microbial Transplant) with Healthy Donor Stool into the jejunum through upper endoscopy.

Group II: Arm 1 Placebo Group 1 Intervention

Placebo Comparator placebo into the jejunum through upper endoscopy.

Who Is Running the Clinical Trial?

Children's Hospital Los Angeles

Lead Sponsor

Trials

257

Recruited

5,075,000+

Paul S. Viviano

Children's Hospital Los Angeles

Chief Executive Officer since 2015

Master of Public Health from UCLA Fielding School of Public Health

Alan S. Wayne

Children's Hospital Los Angeles

Chief Medical Officer since 2023

MD